Kornimate/medical-research-clean huggingface dataset:
vedolizumab study with inflammatory bowel disease patients in germany

Conditions: Inflammatory Bowel Diseases; Treatment

Summary: this study on biologics (vedolizumab/anti-tnf) in the treatment of inflammatory bowel disease (ibd) patients in germany extends the prospective documentation of efficacy in induction and maintenance therapy of anti-tnf to the use of vedolizumab with a particular interest in the construction of a multifactorial model to predict long-term responses and favorable disease outcome or to predict severe side effects caused by therapy with biologics. little data are available on the efficacy and safety of biologics in germany in a real world situation. while the increasing use of anti-tnf-alpha antibodies in ibd-patients the new therapy with vedolizumab opens up new opportunities in ibd-therapy, it may also pose new options and problems in terms of efficacy and predictors of response and potential side effects.

the ibd-patients will be prospectively documented in the bioibd-registry. the diagnosis is made in accordance with current dgvs/ecco uc/cd guidelines. there are the following inclusion and exclusion criteria: inclusion criteria: * ibd-patients (uc/cd) aged 18-80 years at enrollment * written informed consent is given exclusion criteria:lack of adequate documentation possibilities * malignant disease in history * planned surgical intervention there are three subpopulations: 1. ibd-patients (age at enrollment: 18-80 years) receiving a newly introduced vedolizumab (vdz) therapy (n=1.800). a former therapy with other biologics is allowed. more than 30% of these vedolizumab patients will be biologics-naiv. 2. ibd-patients (age at enrollment: 18-80 years) receiving a newly introduced anti-tnf-alpha therapy other than vdz (n=350) in biologics-naiv patients. 3. ibd patients (age at enrollment: 18-80 years) with an early disease (n=350), who were first diagnosed \< 2 years before the start of documentation in the investigator initiated non-interventional study (nis) but have not yet received and are not planned to receive biologics in the near future. patients with ibd on vedolizumab/anti-tnf will be documented in a prospective online documentation form at the participating study sites. in parallel, these sites will also document consecutively early disease ibd patients who were diagnosed less than 2 years previously. these patients will be used as a control group.the data will be documented in an online documentation form. after initial documentation at enrollment and during induction (0, 2, 6 and 14 weeks), follow-up documentation using an abbreviated online follow-up form will be requested every 6 months during the longitudinal investigation. any drug side effects are also captured online on a side effects form.

Eligibility criteria:
inclusion criteria: * ibd-patients (uc/cd) aged 18-80 years at enrollment * written informed consent is given exclusion criteria: * lack of adequate documentation possibilities * malignant disease in history * planned surgical intervention

Keywords: vedolizumab, crohns disease, ulcerative colitis, safety
mesenchymal stem cells in rotator cuff repair

Conditions: Rotator Cuff Tear; Tendon Injuries; Mesenchymal Stem Cell

Summary: randomized clinical study involving 44 patients, evaluating the effect of mesenchymal cells on rotator cuff repair. the primary outcome will be post-operative mri tendon integrity and secondary outcomes clinical assessment by the ucla and american shoulder and elbow surgeons (ases) scales and pain by visual analog scale (vas).

rotator cuff tendinopathy is the main cause of shoulder pain and tear of these tendons affects 20% of the population. although arthroscopic repair leads to satisfactory clinical results, the retear rates varies from 4 to 94%. the main cause of failure after rotator cuff repair is not related to the material used, but to the tissue deficiency and the healing process between the tendon and the bone. after the intervention, the rotator cuff does not restore its original histological characteristics and its fixation occurs through scar tissue with lower biomechanical resistance. there are few clinical studies on the use of mesenchymal cells in rotator cuff repair,, with good results. the investigators will perform a randomized clinical study involving 44 patients, evaluating the effect of mesenchymal cells on rotator cuff repair. the primary outcome will be post-operative mri tendon integrity and secondary outcomes clinical assessment by the ucla and american shoulder and elbow surgeons (ases) scales and pain by visual analog scale (vas).

Eligibility criteria:
inclusion criteria: * full thickness posterosuperior rotator cuff tear, of small or medium size, fully repairable at the time of surgery, regardless of size; * pain and / or decreased shoulder function for at least 3 months, with no improvement with non-surgical treatment; * absence of the following changes in mri: * fat degeneration of the rotator cuff musculature of grade 3 according to the classification of fuchs (presence of equal or superior amount of fat compared to muscle fibers); * absence of the following changes in radiographs * advanced glenohumeral arthrosis (grades 2, 3 and 4 of samilson and prieto * signs of rotator cuff arthropathy, according to seebauer classification; * skeletal maturity; * absence of surgeries or previous fractures in the shoulder in question; * absence of psychiatric illnesses, fibromyalgia or painful pathologies of the cervical spine; * absence of rheumatic diseases or chronic use of corticosteroids; * absence of active or recent infection; * absence of thrombocytopenia, coagulopathies or chronic use of anticoagulants; * absence of vascular or neurological lesions affecting the upper limb; * absence of pregnancy; * clinical non-compensated comorbidities; * chronic use of corticosteroids; * consent to free and informed consent; * live in brazil. exclusion criteria: * visualization during the operative event of one or more of the findings: * irreparable rupture of rotator cuff; * subsecapularis tear involving two thirds or more of its extension; * need to open surgery.
efficacy study of leflunomide to treat juvenile idiopathic arthritis

Conditions: Juvenile Idiopathic Arthritis

Summary: a randomized double blind clinical trial to assess efficacy of leflunomide in treatment of refractory juvenile idiopathic arthritis. patients are randomly divided into two groups. in group 1 leflunomide and in group 2 placebos will be added to conventional treatment for three months. therapeutic responses will be evaluated by acrpedi (american college of rheumatology pediatric) scores every 4 weeks.

30 patients(2-19 y) are randomly divided into two groups. in group 1 leflunomide will be added to conventional treatment, 5-20 mg daily based on weight. in group 2 placebo will be prescribed as same as leflunomide. the course of treatment is 3 months. every 4 weeks acrped 30, 50, 70 should be determined to evaluate therapeutic response. side effects of treatment will be assessed by physical examination and lab tests every 4 weeks.

Eligibility criteria:
inclusion criteria: * jia based on acr criteria * age between 2- 19 y * polyarticular, oligoarticular(\> or = 3 active joints) or extended oligoarticular subtypes * resistance to conventional treatment exclusion criteria: * pregnancy * malignancy * severe active infection * other rheumatic diseases or overlap * alt(alanine transaminase) or bilirubin \> 3 folds * ivig (intravenous immunoglobulin) treatment during last 2 weeks * biologic agents during last 3 months

Keywords: jia leflunomide efficacy
vnotes transvaginal endoscopic surgery versus laparoscopy

Conditions: Salpingectomy

Summary: the feasibility and safety of laparoscopy are no longer to be demonstrated, especially in the field of gynecology, but this technique often causes post-operative pain. new techniques are being developed to overcome the inconvenience of laparoscopy, notably endoscopic transluminal surgery using a natural orifice (notes). it avoids incisions and therefore scarring, and could reduce post-operative pain. it is now developing in the field of transvaginal gynecology (vnotes). this study is the first prospective randomized study comparing the vnotes technique to laparoscopy with postoperative pain assessment for performing a salpingectomy. the hypothesis is that the use of vnotes transvaginal endoscopic surgery would reduce the post-operative pain of patients compared to laparoscopy in the context of a salpingectomy.


Eligibility criteria:
inclusion criteria: * planned surgery procedure : bilateral salpingectomy for outpatient benign indication * patients with free, informed and signed consent exclusion criteria: * disorders leading to an unacceptable risk of postoperative complications sought during the interrogation of the patient (disorders of blood coagulation, disorders of the immune system, progressive diseases ....) * pregnancy or wish for subsequent pregnancy * lactating women * intervention in the context of pelvic cancer * history of uni- or bilateral salpingectomy * contraindication to laparoscopy * history of rectal surgery * endometriosis * pelvic inflammatory disease * genital infection * not eligible for outpatient care * associated surgical procedure planned during the intervention (hysterectomy, sub-urethral strip, prolapse cure ...) * participation in another interventional research related to the gynecological sphere * inability to understand the information given * a person not affiliated to a social security scheme, or deprived of liberty, or under guardianship.

Keywords: vnotes laparoscopy salpingectomy pain
noninvasive support methods for acute respiratory failure of community-acquired pneumonia

Conditions: Community-acquired Pneumonia; Community-Acquired Infections

Summary: this randomized controlled trial aims to compare the efficacy and safety of high-velocity nasal insufflation (hvni) versus standard noninvasive ventilation (niv) in adult patients experiencing acute exacerbations of community-acquired pneumonia (cap) accompanied by hypoxemic respiratory failure. the primary focus is on assessing treatment failure within 48 hours, defined as the need for intubation or death. secondary outcomes include evaluations of gas exchange parameters, patient comfort levels, duration of icu stay, and 28-day mortality rates.

community-acquired pneumonia (cap) is a significant contributor to acute hypoxemic respiratory failure, often necessitating ventilatory support. noninvasive ventilation (niv) has been a conventional approach; however, it can be associated with patient discomfort and varying success rates. high-velocity nasal insufflation (hvni) presents an alternative, delivering heated and humidified oxygen at high flow rates, which may improve oxygenation and patient tolerance. this study is designed as a prospective, randomized, open-label, parallel-group trial involving 100 adult patients diagnosed with cap-induced hypoxemic respiratory failure. participants will be randomly assigned to receive either high-volume non-invasive ventilation (hvni) or non-invasive ventilation (niv). the primary endpoint is treatment failure within 48 hours post-randomization, characterized by the necessity for endotracheal intubation or mortality. secondary endpoints encompass changes in arterial blood gas measurements, patient-reported comfort scores, length of icu stay, and 28-day mortality rates. by systematically comparing hvni and niv in this patient population, the study seeks to determine whether hvni offers a viable and potentially superior alternative to traditional niv methods in managing acute respiratory failure due to cap.

Eligibility criteria:
inclusion criteria: * age ≥ 18 years * clinical and radiographic diagnosis of cap * respiratory failure using partial pressure of oxygen (pao₂) and fraction of inspired oxygen (fio₂) is defined by a partial pressure of oxygen/fraction of inspired oxygen ratio (pao₂/fio₂) ≤ 300 mmhg or a respiratory rate (rr) ≥ 25/min * informed consent obtained exclusion criteria: * hemodynamic instability requiring vasopressors * altered mental status glasgow coma scale (gcs \< 13) * contraindication to niv or hvni (facial trauma, recent upper airway surgery) * do-not-intubate (dni) order

Keywords: cap niv hvni
combination of henagliflozin and continuous subcutaneous insulin infusion in t2dm

Conditions: Type2diabetes

Summary: the multi-center, randomized, controlled clinical trial investigate the efficacy and safety of henagliflozin combined with continuous subcutaneous insulin infusion in newly diagnosed type 2 diabetes

the therapeutic mechanisms of sglt2i and continuous subcutaneous insulin infusion (csii) are complementary. we hypothesized that for newly diagnosed type 2 diabetes (t2dm) with severe hyperglycemia, combination treatment with henagliflozin and csii could achieve glycaemic control in a shorter time, shorten the duration of hospitalization and reduce the total insulin dosage. we also hope to investigate the safety of sglt2i in combination with csii in t2dm based on continuous glucose monitoring system.

Eligibility criteria:
inclusion criteria: 1. diagnosed as type 2 diabetes mellitus, diagnosis time ≤ 2 years 2. age between 20 and 70 years 3. continuous use of any hypoglycemic drug ≤ 7 days within 6 months 4. 9% ≤ hba1c ≤ 14% or fasting blood glucose ≥ 11.1 mmol/l 5. body mass index (bmi) of between 20 and 32 kg/m2 6. be able to understand the contents and methods of this study and sign the informed consent form voluntarily exclusion criteria: 1. diabetic ketosis or ketoacidosis, diabetic hypertonic state, diabetic lactic acidosis and other acute diabetic complications or serious chronic diabetic complications 2. urinary tract infection or/and genital infection with clinical significance, or repeated urinary tract infection or/and genital infection history 3. serious trauma or acute infection occurred within 4 weeks that may affect blood glucose control 4. people with negligent compensatory heart failure (nyha grade iii and iv), unstable angina, stroke or transient ischemic attack, myocardial infarction, serious arrhythmia, cardiac surgery or vascular reconstruction within 6 months 5. serious blood system disease (such as aplastic anemia, myelodysplastic syndrome) or any disease causing hemolysis or erythrocyte instability (such as malaria, hemolytic anemia) 6. severe chronic gastrointestinal diseases, or those who have received treatment that may affect drug absorption (such as gastrointestinal surgery) 7. uncontrolled hyperthyroidism 8. those who have mental or nervous system diseases and are unwilling to communicate or cannot fully understand and cooperate 9. pregnant or lactating women 10. alt\>3.0x uln and/or ast\>3.0x uln and/or tbil\>2.0x uln blood ketone body\>uln egfr\ 3.0x uln

Keywords: continuous subcutaneous insulin infusion sglt2i continuous glucose monitoring type 2 diabetes
a gene therapy study of rp-a501 in male patients with danon disease

Conditions: Danon Disease

Summary: this is a single arm phase 2 trial to evaluate the efficacy and safety of rp-a501, a recombinant adeno-associated virus serotype 9 (aav9) containing the human lysosome-associated membrane protein 2 isoform b (lamp2b) transgene, in male patients with danon disease.

the study is a single arm phase 2 clinical trial to characterize the safety and efficacy of rp-a501, a recombinant adeno-associated serotype 9 (raav9 capsid containing the human lysosome-associated membrane protein 2 isoform b (lamp2b) transgene) in male patients with danon disease. male subjects ≥8 years of age will receive a single intravenous infusion of rp-a501.

Eligibility criteria:
key inclusion criteria: 1. documentation of a pathogenic or likely pathogenic variant of the lamp2 gene. 2. male. 3. age ≥8 years. 4. evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: 1. abnormal thickening of left ventricular wall, 2. left ventricular ejection fraction (lvef) ≥ 50%. 5. new york heart association (nyha) class ii to iii. 6. high sensitivity troponin i (hstni) ≥20% above the upper limit of normal (uln) 7. ability to comply with study procedures including investigational therapy and follow-up evaluations. key exclusion criteria: 1. anti-aav9 neutralizing antibody titer \>1:40. 2. severe heart failure or requirement for advanced therapies. 3. history of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (tia), acute coronary syndrome, myocardial infarction or unstable angina. 4. prior cardiac or other organ (lung, liver, other) transplantation.

Keywords: hypertrophic cardiomyopathy hcm x-linked disease lamp2 pediatric skeletal myopathies cardiomyopathy lysosomal storage disorder left ventricular hypertrophy
biomarkers of kidney function in transplant medicine

Conditions: Acute Kidney Injury

Summary: biomarkers of kidney function in transplant medicine is an international, multicentre, observational, non-interventional study. the project is aimed at monitoring biomarkers of acute kidney dysfunction in deceased organ donors, living organ donors, and organ recipients.

the study is observational, without any changes from the standard care, including only selected laboratory assessments, from standard blood samples collected from the donors/recipients. informed consent will be required from living donors and recipients. the mainly used current criteria of organ acceptability in transplant medicine include urine output and laboratory parameters of acute kidney dysfunction - serum levels of urea and creatinine. literary sources show that these classic criteria of kidney dysfunction develop only with a significant reduction of (glomerular and tubular) kidney functions and return to normal only slowly after the function of the kidneys has been restored. new possibilities of early kidney dysfunction diagnostics are being studied, using more sensitive tests - determination of biomarkers of acute kidney dysfunction. these may serve as decisive criteria for the safe use of organs from so-called marginal donors and identify early serious impairment of kidney function in donors with preserved urine output, without fulfilled criteria of acute kidney injury.

Eligibility criteria:
inclusion criteria: * age 18+ * signed informed consent in case of living organ donors and recipients * fulfillment of all legal requirements for organ harvesting from a deceased donor * fulfillment of all ethical principles of end-of-life patient care * medical suitability of organs for transplant use exclusion criteria: - disapproval of family members with the enrolment of the patient in the study

Keywords: organ donor organ recipient transplant medicine acute kidney injury
effect of peripapillary atrophy to diagnose glaucoma in high myopia

Conditions: Myopia; Refractive Errors; Eye Diseases; Glaucoma, Primary Open Angle; Peripapillary Atrophy

Summary: this study intends to analyze the characteristics between peripapillary retinal nerve fiber layer thickness and peripapillary area in high myopia with or without glaucoma

glaucoma is the leading cause of irreversible blindness in the world. myopia is a risk factor for glaucoma. with the rising prevalence of both myopia and glaucoma in an ageing population, the occurrence of these two ocular conditions in the same patient is likely to increase. it was estimated that there were 163 million people who have high myopia in 2000, and the population with high myopia would increase to almost one billion (9.8% of the world population) worldwide by 2050. there is often a diagnostic challenge to the clinician, since the detection of glaucomatous optic nerve damage in highly myopic eyes is difficult. recently, the subclassification of peripapillary area could potentially be used to differentiate myopic eyes with and without glaucoma according to oct findings. however, the characteristics of peripapillary atrophy have not been fully applied in the diagnosis of high myopia and glaucoma. in view of the above problems, the purpose of this study is to analyze the peripapillary area based on optical coherence tomography and it may be a specific marker for identifying high myopia with primary open angle glaucoma.

Eligibility criteria:
inclusion criteria: 1. age ≥18 years. 2. equivalent spherical ≤-6d or axial length ≥26.5mm. 3. high myopia with primary open angle glaucoma, such as anterior chamber angle is opening, optic rim defect, rnfl loss, etc. 4. the peripapillary region can be accurately defined based on oct. exclusion criteria: (1) exclusion criteria for high myopia group: 1. history of glaucoma and elevated intraocular pressure. 2. history of uveitis or intraocular surgery. 3. history of other retinal optic nerve or related systemic diseases. 4. the examination results are unreliable, such as poor image quality. (2) exclusion criteria for high myopia with primary open angle glaucoma group: 1. others nervous system diseases, such as visual field loss or optic nerve damage. 2. others non-glaucomatous ocular pathologies may affect the visual field or retinal nerve fiber layer status, such as retinal diseases, uveitis, or ocular surgery history.

Keywords: high myopia glaucoma peripapillary atrophy optical coherence tomography
dinutuximab beta at the hus and the toulouse oncopole

Conditions: Neuroblastoma

Summary: in this study the investigators wish to describe the cohort of pediatric patients suffering from neuroblastoma and treated with dinutuximab and to compare the costs and quality of life between the two french centers, the university hospital of strasbourg (hus) and the oncopole of toulouse.


Eligibility criteria:
inclusion criteria: * subjects aged 2 years to 25 years * male or female gender * subjects suffering from neuroblastoma and treated at the hus or at the oncopole center in toulouse during the period from january 1, 1998 to december 31, 2023. * subject (and/or holders of parental authority) not opposing, after information, the reuse of the child's data for the purposes of this research and agreeing to respond to the quality of life survey. exclusion criteria: * patient or parents of the minor having expressed their opposition to participating in the study. * specific clinical forms of the disease, * interfering treatments and associated diseases.

Keywords: neuroblastoma dinutuximab beta dnb